Organoids plus gene editing bring insights into pediatric metabolic disease
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”

Organoids plus gene editing bring insights into pediatric metabolic disease
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
ABI-201 confers protection against age-related macular degeneration
ABI-201 is an engineered AAV.N54 vector that expresses a soluble CD59 variant and a complement 3 (C3) inhibitor fusion protein. ABI-201 was developed by Avirmax Biopharma Inc. to confer photoreceptor protection against retinal damage. The effects of ABI-201 were tested in a sodium iodate-induced retinal damage model in nonhuman primates, a widely used model for testing therapies for age-related macular degeneration (AMD).
Viverita and Boehringer Ingelheim collaborate on cancer target discovery and validation
Viverita Therapeutics Inc. has established a strategic research collaboration with Boehringer Ingelheim Pharma GmbH & Co. KG aimed at accelerating the discovery and validation of novel therapeutic targets for cancer.
WTX-921: An IL-10 Indukine designed to minimize systemic toxicity
Interleukin-10 (IL-10) is an immunoregulatory cytokine produced by a variety of immune cells, including type 1 regulatory T cells and macrophages, as well as nonimmune cells such as epithelial cells and fibroblasts.
Merck Sharp & Dohme patents new IL4I1 inhibitors
Merck Sharp & Dohme LLC has disclosed L-amino acid oxidase (IL4I1; LAAO) inhibitors reported to be useful for the treatment of cancer.
Genprex and University of Pittsburgh sign new agreement to study GPX-002 for diabetes
Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes.
Preclinical results from a new HPV16/18 mRNA-based vaccine presented
Metis Pharmaceuticals Inc. recently presented a novel mRNA-encoded human papillomavirus (HPV) 16/18 therapeutic vaccine for HPV-driven malignancies.

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Conferences

LM-350 shows strong antitumor activity in CDH17-positive models
New compound
Cadherin 17 (CDH17) is a membrane-bound cell adhesion molecule involved in tumor cell proliferation and is selectively overexpressed in several gastrointestinal malignancies, including colorectal cancer, gastric cancer and pancreatic cancer.
Surrozen’s SZN-8141 ameliorates retinopathy in murine models
Ocular
Recent evidence has suggested that the use of norrin mimetics targeting both frizzled-4 (FZD4) and low-density lipoprotein receptor-related protein 5 (LRP5) may be highly effective at modulating retinal vascular leakage. When combined with an anti-VEGF therapy, it was hypothesized to have an...
Beigene’s BGB-A3055 inhibits tumor growth in cancer rodent models
American Association for Cancer Research
Beigene Co. Ltd. has presented data on their anti-CCR8 antibody BGB-A3055 as a cancer immunotherapeutic approach.
PBI-671 induces retinal ganglion cell survival in glaucoma models
Ocular
Glaucoma is a leading cause of blindness and is characterized by the death of retinal ganglion cells (RGCs). Genetic screening data have identified dual leucine zipper kinase (DLK) and leucine zipper kinase (LZK) as important mediators of RGC death.

Cancer

Formosa and Almac Discovery sign license agreement for EGFRxROR1 bispecific ADC
License
Formosa Pharmaceuticals Inc. and Almac Discovery Ltd. have announced a global licensing agreement for development and commercialization of ALM-401, a first-in-class engineered bispecific antibody-drug conjugate (ADC) for the treatment of solid tumors characterized by the dual expression of EGFR and...

Infection

Immunogenicity and efficacy of a bivalent self-amplifying RNA vaccine against yellow fever and Zika
Immune
Researchers from the Access to Advanced Health Institute and collaborating institutions have developed a promising new bivalent vaccine candidate that can protect against both yellow fever and Zika viruses. The study demonstrates that the vaccine elicits robust humoral and cellular immune responses...

Key genes and educated natural killer cells keep HIV in remission
Immune
Scientists at Institut Pasteur have gained new insights into how some people control HIV-1 replication after interruption of antiretroviral treatment (ART). The investigators found a fingerprint involved in long-term viral remission.
Nuvobio divulges new peptides to treat C. neoformans infection
Patents
Nuvobio Corp. has synthesized peptides reported to be useful for the treatment of Cryptococcus neoformans infection.

Neurology/psychiatric

Solve FSHD supports Armatus Bio drug for facioscapulohumeral muscular dystrophy
Financings
Armatus Bio Inc.’s development of ARM-201, an AAV-delivered microRNA therapy for facioscapulohumeral muscular dystrophy (FSHD), has been boosted by a $3 million investment by Solve FSHD.
Alchemab and Lilly sign licensing agreement for ATLX-1282
Deals and M&A
Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
A SUMO1 peptide that might stop α-synuclein aggregation before it begins
Peptide
Abnormal aggregation of α-synuclein is thought to contribute to the pathology of Parkinson’s disease, the second most frequent neurodegenerative disorder. Therefore, developing drugs that prevent such aggregation could be effective for slowing or even preventing the disease.
New tachykinin NK3 receptor antagonists disclosed in Kallyope patent
Patents
Kallyope has divulged compounds acting as tachykinin NK3 receptor antagonists reported to be useful for the treatment of migraine.
Unravel seeks Colombian approval to study RVL-001 for Rett and Pitt-Hopkins syndromes
Regulatory
Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.

Endocrine/metabolic

Mammoth nominates development candidate for FCS and severe hypertriglyceridemia
Cardiovascular
Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.

Biomarkers

Variants in MED16 gene cause intellectual disability MEDopathy
The Mediator complex is a system that regulates protein-coding gene transcription, where mediator of RNA polymerase II transcription subunit 16 (MED16) is a subunit belonging to this system. Pathogenic genetic variants in Mediator complex subunits usually lead to neurodevelopmental and...

Plasma ANG2 predicts poor outcomes after traumatic brain injury
Conferences
Investigators from the University of Pennsylvania have presented data regarding the relationship of angiopoietin-2 (ANG2) and its prognostic impact on traumatic brain injury (TBI). The ANG2 plasmatic levels were measured in patients with TBI (n=362), orthopedic injury controls (n=89) and healthy...
Ignite Biomedical, Liquid Biosciences discover new biomarkers for substance use disorder
Substance use and poisoning
Ignite Biomedical Inc. and Liquid Biosciences have announced the discovery of biomarkers to diagnose substance use disorder (SUD). The set of biomarkers includes some that have not previously been associated with addiction and spans all 6 SUDs (opioid, cocaine, methamphetamine, alcohol, nicotine...

Gastrointestinal

Plant compound PEITC may mitigate both oxidative stress and inflammation in ulcerative colitis
Ulcerative colitis, one type of chronic inflammatory bowel disease, is driven by a form of programmed cell death called pyroptosis.
Equillium advancing new AhR modulator
Inflammatory
Equillium Inc. is advancing EQ-504, a potent aryl hydrocarbon receptor (AhR) modulator acquired through the acquisition of Ariagen Inc., a company that was majority owned by Equillium’s largest investor, Decheng Capital.
Bristol Myers Squibb divulges new RIPK1 inhibitors
Patents
Bristol Myers Squibb Co. has synthesized receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of inflammatory bowel disease, psoriasis, rheumatoid arthritis, nonalcoholic or...
CREG1 protects against acetaminophen-induced liver damage by regulating autophagy
Substance use and poisoning
Acetaminophen (APAP), although recognized as a safe and effective antipyretic and analgesic drug, can lead to acute liver failure if taken in excess.
PVT-401 achieves milestone under Parvus and Abbvie inflammatory bowel disease collaboration
Nanotech
Parvus Therapeutics Inc. has announced the achievement of a milestone under its collaboration with Abbvie Inc. to develop treatments for inflammatory bowel disease (IBD). PVT-401, a novel peptide-major histocompatibility complex (pMHC)...
Germline variants’ impact on pan-cancer proteome
Omics
A large-scale study has revealed the impact of germline variants on proteins in 10 cancer types. Scientists from the National Cancer Institute’s Clinical Proteomic Tumor Analysis Consortium (CPTAC) conducted a precision proteogenomic analysis in a...

podcast microphone, sound waves on purple background Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.

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